Glossary

Adeno-associated virus
A type of virus that occurs in nature, but is not believed to cause illness in humans. It requires another virus, such as an adenovirus or herpesvirus, in order to reproduce. It is currently being researched in gene transfer therapy as part of a therapeutic vector to introduce functional genes into cells.
Cell
The fundamental, structural, and functional unit of living organisms. Gene transfer therapy research is evaluating whether it can target specific cells in order to evaluate the safety risks and impact with the body.
DNA (deoxyribonucleic acid)
The molecular basis of heredity in humans. Mutations in DNA can cause genetic conditions like hemophilia.
Factor VIII
A blood-clotting protein encoded by the F8 gene. Coagulation factors are essential in the formation of stable blood clots. Factor VIII is missing or not working in people with hemophilia A. Research is ongoing to determine if gene therapy may help people with hemophilia A produce their own factor VIII.
Factor IX
A blood-clotting protein encoded by the F9 gene. Coagulation factors are essential in the formation of stable blood clots. Factor IX is missing or not working in people with hemophilia B. Research is ongoing to determine if gene therapy may help people with hemophilia B produce their own factor IX.
Gene
A part of a DNA molecule that tells your body how to make a protein. A mutation within certain genes can lead to lacking or missing proteins, which can lead to genetic conditions. Gene therapy aims to help the body produce the protein it needs.
Gene Transfer Therapy
One method of gene therapy currently being explored in clinical trials. This approach aims to introduce a working gene into the body to evaluate whether it can produce a missing or deficient protein.
Genetics
The study of genes and their heredity.
Mutation
A change in the structure of a gene that then becomes a non-working form, which may cause problems (or damage) and may be passed along to future generations. It is caused by the alteration, deletion, insertion, or rearrangement of parts of DNA.
Neutralized virus
The shell of a virus without any viral DNA inside. In gene transfer therapy, the shell is used to protect and transport the functional gene DNA.
Protein
A substance created by a gene that performs various biological functions. Examples include enzymes, hormones, and antibodies. Proteins also make up our bones, organs, and tissues.
Therapeutic vector
A vehicle used to deliver the working, or functional, gene into the body.
Vector shedding
The release of vector particles from the body of the person who has received gene therapy. Scientists are studying clearance of these materials from the body and whether they could pose any risk to others or to the environment.
Working gene, or functional gene
A piece of DNA designed by scientists to make a desired protein. It is intended to supplement the functionality of a mutated or missing gene to target the specific needs of different genetic conditions.

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